Financial Data and Key Metrics Changes - The company reported revenues of $134 million for Q2 2022 and $276 million for the first half of the year, showing an increase compared to the same periods last year [16] - SPINRAZA's global sales were $431 million for Q2 and $904 million year-to-date, resulting in $60 million and $113 million in royalty revenue for the corresponding periods [17] - Cash and investments remained flat at $2 billion from Q1 to Q2 [17] - Non-GAAP operating expenses increased due to higher R&D expenses, while SG&A expenses decreased year-over-year [23][24] - The company anticipates Q3 revenues to be similar to Q2, with R&D expenses projected to increase by 25% to 30% compared to last year [25][26] Business Line Data and Key Metrics Changes - The company is advancing its late-stage pipeline, with positive results from the Phase III NEUROTTRansform study of eplontersen in patients with TTR polyneuropathy [8][30] - Enrollment in the BALANCE study of olezarsen for familial chylomicronemia syndrome has been completed, with data expected next year [11][33] - The company reported positive data from six mid-stage programs, with plans to advance two new medicines into Phase III studies next year [13] Market Data and Key Metrics Changes - SPINRAZA sales in the US stabilized year-to-date, with a decrease in discontinuations during the quarter [18] - The company expects SPINRAZA to return to growth as positive trends in the US market are observed [18] - The Lp(a) HORIZON cardiovascular outcome study of pelacarsen has enrolled over 8,000 patients, solidifying the company's first-mover advantage [12][40] Company Strategy and Development Direction - The company aims to deliver transformational medicines to the market, with a focus on expanding its Phase III pipeline to at least eight medicines across ten indications [14][46] - The partnership with AstraZeneca is crucial for the global go-to-market strategy for eplontersen and other products [9][76] - The company is also focused on advancing new chemistries and expanding its LICA platform beyond the liver [47] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the positive results from ongoing studies and the potential for eplontersen and tofersen to enter the market soon [10][48] - The company is well-positioned to maximize value for patients and shareholders through its strategic investments and pipeline advancements [14][27] - Management highlighted the importance of generating robust clinical data to support the competitive positioning of its products in the market [111][114] Other Important Information - The NDA for tofersen is under priority review with the FDA, with a PDUFA date set for January 25, 2023 [10][36] - The company is preparing for additional studies related to eplontersen as part of its life cycle management strategy [70] Q&A Session Summary Question: What do you hope to show at the upcoming presentation at the ISA meeting regarding Eplontersen? - Management expressed excitement about sharing the efficacy, safety, and tolerability data at the ISA meeting, emphasizing the importance of the primary and secondary endpoints [50][51] Question: Will you consider filing any of your other neuro or ALS programs with NFL as a surrogate endpoint? - Management indicated that while biomarkers are important, they do not plan to file based solely on biomarkers but will use them as part of a robust study design [62][64] Question: Can you provide an update on the HBV program with GSK? - Management highlighted encouraging data from GSK's Bepirovirsen and the intention to move into Phase 3 development [84][85] Question: What is the differentiation of your Angelman program from other therapies? - Management noted that their drug targets the antisense transcript and aims to be the best molecule in the space, with a focus on safety and target engagement [102][104] Question: Can you confirm if you have done an interim analysis on cardio transform before expanding to 1,000 patients? - Management clarified that the decision to expand the study was based on demographics rather than event rates, emphasizing the importance of a robust data set [131][132]

IONIS | --- | --- | --- | --- | --- | |----------------|-------|-------|-------|-------| | | | | | | | August 9, 2022 | | | | | | Nasdaq: IONS | | | | | | | | | | | On Today's Earnings Call 2 Brett Monia, Ph.D. Chief Executive Officer Beth Hougen Chief Financial Officer Richard Geary, Ph.D. Executive Vice President, Development Eric Swayze, Ph.D. Executive Vice President, Research Eugene Schneider, M.D. Executive Vice President, Chief Clinical Development Officer Onaiza Cadoret Executive Vice President, Chi ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Delaware 33-0336973 2855 Gazelle Court, Carlsbad, California 92010 (Address of Principal Executive Offices) (Zip Code) 760-931-9200 For the Quarterly Period Ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file nu ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q1 2022 Earnings Conference Call May 4, 2022 11:30 AM ET Company Participants Brett Monia - Founder, CEO & Director Elizabeth Hougen - EVP, Finance & CFO Eugene Schneider - EVP & Chief Clinical Development Officer Eric Swayze - EVP, Research Onaiza Cadoret - EVP and Chief Product Strategy & Operations Officer Conference Call Participants Yanan Zhu - Wells Fargo Securities Brendan Smith - Cowen and Company Luca Issi - RBC Capital Markets Jessica Fye - JPMorgan Chase ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended March 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) (State or other jurisdiction of i ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q4 2021 Results Earnings Conference Call February 24, 2022 11:30 AM ET Company Participants Jennifer Capuzelo - Director of Investor Relation Brett Monia - Founder & CEO Beth Hougen - Chief Financial Officer Richard Geary - Executive Vice President & Chief Development Officer Eric Swayze - Executive Vice President of Research Onaiza Cadoret-Manier - Executive Vice President & Chief Product Strategy & Operations Officer Conference Call Participants Gary Nachman - BMO ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 FORM 10-K ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ___________ to ___________ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or other ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q3 2021 Earnings Conference Call November 3, 2021 11:30 AM ET Company Participants Jennifer Capuzela - Director of Investor Relation Brett Monia - Founder, CEO & Director Beth Hougen - Chief Financial Officer Richard Geary - EVP & Chief Development Officer Onaiza Cadoret-Manier - EVP and Chief Corporate Development & Commercial Officer Eric Swayze - EVP of Research Conference Call Participants Yale Jen - Laidlaw & Co Chi Meng Fong - BofA Securities Yanan Zhu - Wells ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, DC 20549 Form 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Quarterly Period Ended September 30, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF SECURITIES EXCHANGE ACT OF 1934 For the transition period from _____ to _____ Commission file number 000-19125 Ionis Pharmaceuticals, Inc. (Exact name of Registrant as specified in its charter) Delaware 33-0336973 (State or ...

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q2 2021 Earnings Conference Call August 4, 2021 11:30 AM ET Company Participants David Nakasone - IR Brett Monia - Founder, CEO & Director Elizabeth Hougen - EVP, Finance & CFO Richard Geary - EVP & Chief Development Officer Eric Swayze - EVP, Research Onaiza Cadoret-Manier - EVP & Chief Corporate Development & Commercial Officer Conference Call Participants Jason Gerberry - Bank of America Merrill Lynch Luca Issi - RBC Capital Markets Brendan Smith - Cowen and Comp ...